GeneTherapy
Springer Nature's monthly journal publishing high-quality research and insights in gene and stem cel
Gene Therapy covers both the research and clinical applications of the new genetic therapy techniques currently being developed. Over the last decade, gene therapy protocols have entered clinical trials in increasing numbers and as they cover a wide spectrum of diseases, these studies promise to unite the diverse organ-based specialties into which modern medicine has become divided. Gene Therapy c
21/07/2023
The Clarivate 2022 Impact Factors are out, and GeneTherapy went up from 4.184 to 5.1!
The editorial team couldn't be more delighted. We thank all our , and for their continued support!
20/07/2023
Trending now is a review discussing the outcomes of in focusing on the types of cells and functional effects of host immune responses on graft survival and differentiation. Join our here now➡️https://www.nature.com/articles/s41434-019-0077-4
📸: ©iStock
17/07/2023
Online at Nature, a splice switching , atipeksen, administered intrathecally three years in a pediatric patient, was well tolerated with notable clinical improvements in disease progression.
https://www.nature.com/articles/s41586-023-06277-0
10/07/2023
New issue alert ➡️https://www.nature.com/gt/volumes/30/issues/6
📸: Horizontal brain section of an animal four weeks post intravitreal injection of -SYN-eGFP from a paper by Bart Nieuwenhuis et al.
07/07/2023
Bilateral intravitreal injection of triple phosphodegron mutant capsid is associated with increased local and systemic immune activation
➡️https://www.nature.com/articles/s41434-023-00409-x
🧪: University of Cambridge, Guy's and St Thomas' NHS Foundation Trust, Moorfields Eye Hospital NHS Foundation Trust, UCL Institute of Ophthalmology & The University of Melbourne
📸: Retinal whole-mount
06/07/2023
Trending now is an Orchard Therapeutics review exploring the rationale, clinical application, technological advancement, commercialisation and challenges of targeting . oin our here now➡️https://www.nature.com/articles/s41434-021-00261-x
📸:©iStock
20/06/2023
Reported at , favourable safety and efficacy in the first four patients with in the RUBY trial treated with EDIT-301, the world's first CRISPR/CA12 by Editas Medicine.
20/06/2023
Trending now is a 2004 out of Vanderbilt University discussing the therapeutic potential of . For more, join our here now ➡️ https://www.nature.com/articles/3302356
📸: ©iStock
19/06/2023
Online now, mediated of the SMn2B/- gene in -derived motor neurons in vitro and in a neonatal SMA mouse model restores SMN protein levels. Read here ➡️https://www.nature.com/articles/s41434-023-00406-0
🧪: RJYanezMunoz's lab at Royal Holloway, University of London
📸: SMA iPSC-derived MNs
11/05/2023
This week the world's first vaccine, by GSK targeting for use in adults 60 and older has been approved and granted Priority Review designation by U.S. Food and Drug Administration.
📸: ©iStock
10/05/2023
Online now the translational journey highlighting cardiac safety findings of cutting-edge , ,for ⬇️
https://www.nature.com/articles/s41434-023-00401-5
📝 :Novartis, Children's Health Ireland, UI Health and Inova Children's researchers
📸: ©iStock
09/05/2023
Trending now, intracerebral injection of vector-mediated of SOD1 and GPx1 reduces gp120-induced neurotoxicity 24 weeks post-administration. For more, join here now➡️https://www.nature.com/articles/3303030
🧪: Thomas Jefferson University
📸: ©iStock
03/04/2023
Online now, mediated of full-length rhodopsin gene retains noncoding genomic elements and improves rod photoreceptor structural and functional recovery in animal models. here➡️https://www.nature.com/articles/s41434-023-00394-1
Work by: UNC School of Medicine
📸: Retina Image Bank
01/04/2023
Trending now, injection of -mediated HGF reduces astrogliosis in the motor cortex of animal models. For more, join our here now➡️https://nature.com/articles/s41434-023-00383-4
Work by Northwestern University Helixmith Co. & Seoul National University
📸: ©iStock
30/03/2023
This week, Cellusion Co. announced its -derived endothelial , CLS001, has been transplanted into the first patient in a first-in-human trial.
📸:©iStock
18/03/2023
Trending now, Imperial College London, UCL & ImuThes Ltd scientists demonstrate long-term in vivo expression of non-viral episomal pDNA vectors containing a S/MAR attachment region and liver-specific AAT promoter. Join our here for more➡️https://www.nature.com/articles/gt2008113
16/03/2023
Online Oxford Academic (Oxford University Press) scientists University of Bristol report of a -associated variant of the BPIFB4 gene reverses the decline of performance in animal models.
📸: ©iStock
https://doi.org/10.1093/cvr/cvad008
14/03/2023
Online now, injection of -mediated HGF reduces astrogliosis in the motor cortex of animal models. Read here now➡️https://www.nature.com/articles/s41434-023-00383-4
Work by Northwestern University Helixmith Co., Seoul National University
📸: ©iStock
25/02/2023
Trending again is the review article out of Wits - University of the Witwatersrand exploring self-amplifying and therapeutic candidates for infectious diseases. Join our here now➡️https://www.nature.com/articles/s41434-020-00204-y
📷: ©iStock
23/02/2023
New issue out now! ➡️https://www.nature.com/gt/volumes/30/issues/1-2
On the cover, Purkinje cells transduced by AAV-GFP following intracisternal
21/02/2023
Online now, Huazhong University researchers use -based homology-directed repair of the MYBPC3 gene to rescue phenotypes in an animal model. Read here now➡️https://www.nature.com/articles/s41434-023-00384-3
📷: ©iStock
20/02/2023
Online , and discuss , its impacts on government spending, and the sustainability of expenditures in the public health systems of low-to-middle income countries
📷:©iStock
https://www.nytimes.com/2023/01/24/health/gene-therapies-cost-zolgensma.html
18/02/2023
Online now, the rate of -based deletion of a GAA repeat in the FXN gene in a animal model does not sufficiently increase FXN mRNA levels in the heart. Read more here⬇️
https://www.nature.com/articles/s41434-023-00387-0
Work by: Université Laval
📷 : ©iStock
16/02/2023
Trending now is the 2020 review, out of Üsküdar Üniversitesi on and diagnostic and therapeutic approaches targeted to .
Join our here now⬇️ https://www.nature.com/articles/s41434-020-00209-7
📷:©iStock
11/02/2023
Online now, intra-muscular administration of .2FF antibodies in animal models leads to passive maternal transfer providing sterilising immunity in progeny. Read by University of Guelph & Avamab Pharma ➡️https://www.nature.com/articles/s41434-023-00385-2
📷: ©iStock
09/02/2023
This week University of Pittsburgh researchers propose a Congruence Analysis for Model Organisms framework that quantifies and visualises inter-species concordance/discordance of molecular mechanisms and pathways mimicked by model organisms at PNAS
https://www.pnas.org/doi/10.1073/pnas.2202584120
📷: NHGRI
07/02/2023
Trending now is the 2020 , out of Shanghai Jiao Tong University on the historical developments and future perspectives of research in . Our are here now➡️https://www.nature.com/articles/s41434-020-0163-7
📷: ©iStock
04/02/2023
This week in the European Journal of Human Genetics - EJHG, a Macquarie University & CSIRO survey on in Australia reveals the need for systemic interventions to balance the safeguarding and sharing of .
https://www.nature.com/articles/s41431-022-01273-w
📷:©iStock
02/02/2023
Trending now is the 2007 , out of University of Pittsburgh, demonstrating an increase in anti- antibodies 12 weeks post direct of AAV-TNFRII-Fc capsids in a large animal model. Join our here for more➡️https://www.nature.com/articles/3303020
📷: ©iStock
31/01/2023
Online now, Universitätsklinikum Hamburg-Eppendorf - UKE rresearchers assess the efficacy and cytotoxicity of lectin-based -CARs as an alternative to antibody-based targeting cancer-associated glycan-structures in . 🔓 here➡️https://www.nature.com/articles/s41434-022-00374-x
📷: luismmolina